SEC Filings

8-K
AVEXIS, INC. filed this Form 8-K on 06/14/2017
Entire Document
 

Exhibit 99.1

 

 

Media Inquiries:

Lauren Barbiero

W2O Group

646-564-2156

lbarbiero@w2ogroup.com

 

Investor Inquiries:

Jim Goff

AveXis, Inc.

650-862-4134

jgoff@avexis.com

 

AveXis Announces Alignment with FDA on GMP Commercial Manufacturing Process for AVXS-101

 

— Agency is supportive of proposed analytical methods and comparability protocol —

 

—  AveXis anticipates initiating U.S. SMA Type 1 and Type 2 trials in Q3 2017 with product from new process —

 

—  AveXis manufacturing facility is fully operational for GMP product production —

 

— Conference call and webcast today at 4:30 p.m. EDT —

 

Chicago, Ill. (June 14, 2017) — AveXis, Inc. (NASDAQ: AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced alignment with the U.S. Food and Drug Administration (FDA) on the company’s Good Manufacturing Practice (GMP) commercial manufacturing process for AVXS-101 following the receipt of minutes from the Type B Chemistry Manufacturing and Controls (CMC) meeting. This alignment includes support for the proposed commercial manufacturing process, the proposed analytical methods and corresponding qualification and validation plans — inclusive of key release assays such as potency, purity and identity — and the proposed comparability protocol, which helps assess how similar the product derived from the GMP process is to the original product used in the Phase 1 trial of AVXS-101 in patients with spinal muscular atrophy (SMA) Type 1.

 

Key components of the scalable, GMP commercial manufacturing process are as follows:

·                  AveXis will continue to utilize HEK293 cells and an adherent cell line.

·                  The commercial-scale GMP process will not include the use of Hyperstacks for AAV9 vector production.

 

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